Study of Inhaled RCT1100 in Adults With PCD Caused by Pathogenic Mutations in the DNAI1 Gene to Measure Mucociliary Clearance

NCT ID: NCT06633757

Last Updated: 2025-10-20

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE1
• Total Enrollment: 12 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-10-22
• Study Completion Date: 2025-12-30

Brief Summary

This is a multi-dose study with RCT1100 and is designed to provide safety, tolerability and preliminary efficacy data for future clinical studies.

Detailed Description

The primary objective of this study is to determine the impact of multiple doses of inhaled RCT1100, administered via nebulizer, on MCC with adult participants with Primary Ciliary Dyskinesia caused by pathogenic mutations in the DNAI1 Gene.

Conditions

Primary Ciliary Dyskinesia (PCD)

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

PCD Participants

RCT1100 mRNA therapy supplied to eligible participants with Primary Ciliary Dyskinesia caused by disease-causing mutations in the DNAI1 gene

Group Type: EXPERIMENTAL

RCT1100

Intervention Type: DRUG

mRNA therapy supplied as varying dose strengths administered via oral inhalation using nebulizer

Interventions

RCT1100

mRNA therapy supplied as varying dose strengths administered via oral inhalation using nebulizer

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• The participant is a male or female, 18 to 75 years of age, inclusive, at the time of consent.
• Participant has disease-causing (pathogenic and/or likely pathogenic) mutations in the DNAI1 gene.
• The participant has a percent predicted forced expiratory volume in 1 second (FEV1pp) of at least 40% predicted.

Exclusion Criteria

• History or presence of clinically significant medical, surgical, clinical laboratory, or psychiatric condition or disease.
• History of cancer, with exception of adequately treated basal cell or squamous cell carcinoma of the skin.
• Predisposition to bleeding or clinically meaningful hemorrhagic event in the 12 months prior
• Medically significant hemoptysis.
• Anticoagulation therapy for the treatment of a pulmonary embolus or has had a pulmonary embolus in the last 6 months of screening.
• Active tuberculosis infection.
• 12-lead ECG with QT interval >450 msec (or >480 msec for BBB)
• Laboratory abnormalities in clinical laboratory tests at screening:

1. Serum creatinine level

2. Total bilirubin, aspartate aminotransferase or alanine aminotransferase values

3. Hematological or coagulation values outside the normal reference range

• Any medical history of disease that has the potential to cause a rise in total bilirubin over the ULN.
• COVID-19 infection within 4 weeks of Screening or receipt of COVID-19 vaccine within 2 weeks prior to first dose of RCT1100.
• Receipt of vaccine with live virus, attenuated live virus, or live viral components within 2 weeks prior to first dose of RCT1100 or to receive these vaccines during treatment or within 8 weeks of completion of study treatment.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 75 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

ReCode Therapeutics

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

John Matthews, MBBS, MCRP, PhD

Role: STUDY_CHAIR

ReCode Therapeutics, Inc.

Heymut Omran, MD

Role: PRINCIPAL_INVESTIGATOR

University Hospital Muenster

Kim G Nielsen, Dr Med Sci

Role: PRINCIPAL_INVESTIGATOR

Rigshospitalet, Denmark

Locations

UNC

Chapel Hill, North Carolina, United States

Copenhagen University Hospital - Rigshospitalet

Copenhagen, , Denmark

Münster University Hospital, Albert-Schweitzer-Campus 1

Münster, North Rhine-Westphalia, Germany

Countries

United States; Denmark; Germany

Other Identifiers

RCT1100-103

Identifier Type: -

Identifier Source: org_study_id